公开(公告)号：US20240318187A1

公开(公告)日：2024-09-26

申请号：US18477788

申请日：2023-09-29

Applicant: ModernaTX, Inc.

Inventor： Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Staci SABNIS ,  William BUTCHER

IPC: C12N15/67 ,  A61K31/7115 ,  A61K48/00

CPC classification number: C12N15/67 ,  A61K31/7115 ,  A61K48/0066 ,  C12N2800/22

Abstract: The invention related to polyribonucleotides comprising an open reading frame of linked nucleosides encoding a polypeptide of interest (e.g., a therapeutic polypeptide), isoforms thereof, functional fragments thereof, and fusion proteins comprising the polypeptide. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the invention provides sequence-optimized polyribonucleotides comprising nucleotides encoding the sequence of the polypeptide of interest, or sequence having high sequence identity with those sequence optimized polyribonucleotides.

公开(公告)号：US20190300906A1

公开(公告)日：2019-10-03

申请号：US16302341

申请日：2017-05-18

Applicant: MODERNATX, INC.

Inventor： Paolo MARTINI ,  Stephen HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain McFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Ding AN ,  Staci SABNIS

IPC: C12N15/88 ,  C07K14/14 ,  C12N9/12 ,  B82Y5/00 ,  G01N33/68 ,  A61K9/51

Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).

公开(公告)号：US20190000933A1

公开(公告)日：2019-01-03

申请号：US16110833

申请日：2018-08-23

Applicant: ModernaTX, Inc.

Inventor： Paolo MARTINI ,  Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Xuling ZHU ,  Lin Tung GUEY ,  Staci SABNIS

IPC: A61K38/47 ,  A61K9/51 ,  A61P3/00

Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.